The UK Cystic Fibrosis Gene Therapy Consortium
Who are we
Profiles of the Consortium Strategy Group
Frequently Asked Questions
All Trials Registered Initiative
About this Website
Research Assistant in Genome Editing and Gene Therapy (Oxford)
The Cystic Fibrosis Trust
Just Gene Therapy
What is Cystic Fibrosis?
History of Cystic Fibrosis
Discovery of the CFTR Gene
What causes Cystic Fibrosis?
CFTR Protein Structure
Introduction to Gene Therapy
Why use Gene Therapy to Treat CF?
Other CF Gene Therapy Groups
Successful Applications of Gene Therapy
CF Gene Therapy Clinical Trials
GL67A pGM169 - Our first clinical trial product
The Tracking Study
The Run In Study
Single Dose Clinical Trial (2009-2011)
Multi Dose Clinical Trial (2012-2014)
Multi Dose Clinical Trial Protocol
Public Meeting, 30 May 2015
Nonviral Vector Development
Aerosol Delivery of Gene Transfer Vectors
Lentiviral Vector Development
A novel mixing device for the reproducible generation of nonviral gene therapy formulations.
Davies LA et al., Biotechniques. 2010 Sep;49(3):666-8. doi: 10.2144/000113498.
In vivo imaging of gene transfer to the respiratory tract.
Griesenbach U et al., Biomaterials. 2008 Apr;29(10):1533-40. Epub 2007 Dec 21.
Using magnetic forces to enhance non-viral gene transfer to airway epithelium in vivo.
Xenariou S et al., Gene Ther. 2006 Nov;13(21):1545-52. Epub 2006 Jun 1.
Bactofection of lung epithelial cells in vitro and in vivo using a genetically modified Escherichia coli.
Larsen MD et al., Gene Ther. 2008 Mar;15(6):434-42. doi: 10.1038/sj.gt.3303090. Epub 2008 Jan 24.
Assessment of the nuclear pore dilating agent trans-cyclohexane-1,2-diol in differentiated airway epithelium.
Griesenbach U et al., J Gene Med. 2012 Jul;14(7):491-500. doi: 10.1002/jgm.2643.
Rapid identification of novel functional promoters for gene therapy.
Pringle IA et al., J Mol Med (Berl). 2012 Dec;90(12):1487-96. doi: 10.1007/s00109-012-0928-6. Epub 2012 Jul 6.
Low-frequency ultrasound increases non-viral gene transfer to the mouse lung.
Xenariou S et al., Acta Biochim Biophys Sin (Shanghai). 2010 Jan;42(1):45-51.
Optimizing aerosol gene delivery and expression in the ovine lung.
McLachlan G et al., Mol Ther. 2007 Feb;15(2):348-54.
The role of doxorubicin in non-viral gene transfer in the lung.
Griesenbach U et al., Biomaterials. 2009 Apr;30(10):1971-7. doi: 10.1016/j.biomaterials.2008.12.037. Epub 2009 Jan 18.
Use of ultrasound to enhance nonviral lung gene transfer in vivo.
Xenariou S et al., Gene Ther. 2007 May;14(9):768-74. Epub 2007 Feb 15.
Detection of CFTR transgene mRNA expression in respiratory epithelium isolated from the murine nasal cavity.
Holder E et al., J Gene Med. 2010 Jan;12(1):55-63. doi: 10.1002/jgm.1413.
Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung.
Alton EW et al., Gene Ther. 2014 Jan;21(1):89-95. doi: 10.1038/gt.2013.61. Epub 2013 Nov 7.
Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo.
Griesenbach U et al., Respir Res. 2006 Feb 15;7:26.
The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways.
Griesenbach U et al., Biomaterials. 2010 Mar;31(9):2665-72. doi: 10.1016/j.biomaterials.2009.12.005. Epub 2009 Dec 21.
Secreted Gaussia luciferase as a sensitive reporter gene for in vivo and ex vivo studies of airway gene transfer.
Griesenbach U et al., Biomaterials. 2011 Apr;32(10):2614-24. doi: 10.1016/j.biomaterials.2010.12.001. Epub 2011 Jan 15.
Pre-clinical evaluation of three non-viral gene transfer agents for cystic fibrosis after aerosol delivery to the ovine lung.
McLachlan G et al., Gene Ther. 2011 Oct;18(10):996-1005. doi: 10.1038/gt.2011.55. Epub 2011 Apr 21.
The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep.
Alton EW et al., Biomaterials. 2013 Dec;34(38):10267-77. doi: 10.1016/j.biomaterials.2013.09.023. Epub 2013 Oct 3.
Limitations of the murine nose in the development of nonviral airway gene transfer.
Griesenbach U et al., Am J Respir Cell Mol Biol. 2010 Jul;43(1):46-54. doi: 10.1165/rcmb.2009-0075OC. Epub 2009 Jul 31.
A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis.
Alton EW et al., Efficacy and Mechanism Evaluation (2016) Volume: 3 Issue: 5
Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.
Alton EW et al., Lancet Respir Med. 2015 Sep;3(9):684-91. doi: 10.1016/S2213-2600(15)00245-3. Epub 2015 Jul 3.
A Novel Mixing Device for the Reproducible Manufacture of Non-Viral Gene Therapy Formulations.
Davies LA et al.,The American Society of Gene Therapy Annual Conference (2009)
The nuclear pore dilating Agent TCHD increases gene transfer into differentiated airway epithelium ex vivo, but has no efect in vivo.
Griesenbach U et al.,The American Society of Gene and Cell Therapy Annual Conference (2011)
Optimisation of Aerosol Delivery of Lipid/DNA Complexes for Clinical Studies.
Davies LA et al.,The American Society of Gene Therapy Annual Conference (2008)
Secreted Gaussia Luciferase Is a More Sensitive Reporter Than Firefly Luciferase for Non- Viral Gene Transfer to Airway Epithelium Ex Vivo and In Vivo.
Griesenbach U et al.,The American Society of Gene Therapy Annual Conference (2009)
Aerosol Characteristics of DNA/lipid Formulations for Gene Therapy Clinical Studies.
Gill DR et al.,The North American Cystic Fibrosis Conference (2008)
Mutliple Doses of Lipid Mediated Gene Therapy Nebulised to the Mouse Lung Show Robust and Sustained CFTR Expression.
Hyde SC et al.,The North American Cystic Fibrosis Conference (2011)
The Effect of pDNA Quality on Gene Transfer Outcome In Vivo.
Bazzani RP et al.,The American Society of Gene Therapy Annual Conference (2009)
Safety and expression of a single dose of lipidmediated CFTR gene therapy to the upper and lower airways of patients with Cystic Fibrosis.
Davies G et al.,British Thoracic Society Winter Meeting (2011)
Towards Gene Therapy for Cystic Fibrosis: Bio-Distribution of GL67A/pGM169 DNA and mRNA Following Aerosol Delivery to the Mouse Lung.
Pringle IA et al.,The American Society of Gene Therapy Annual Conference (2008)
A phase IIb Double-Blind Placebo-Controlled Trial of Non-Viral Gene Transfer for Cystic Fibrosis.
Pringle IA et al.,The American Society of Gene and Cell Therapy Annual Conference (2014)
Repeat Administration of Gl67A/pGM169 Is Feasible, Safe, and Produces Endogenous Levels of CFTR Expression After 12 Doses.
Alton EW et al.,British Thoracic Society Winter Meeting (2012)
Cumulative CFTR expression following repeated aerosol delivery of non-viral pGM169/GL67A formulation to mouse lung.
Sumner-Jones SG et al.,The European Society of Gene and Cell Therapy (2012)
Repeated Administration of the Non-Viral Gene Transfer Agent pGM169/GL67A Does Not Induce Anti-CFTR or Anti-Plasmid Immunoresponses.
Griesenbach U et al.,The North American Cystic Fibrosis Conference (2015)
Lentivirus Animation (2011): Basic description of Lentivirus Pseudotyping.
A frozen vial of GL67A (left) and a frozen vial of pGM169 plasmid DNA (right)
Light microscope image of a human airway liquid interface cultures. Dark patches are mucous.
A cake that only some of us got to enjoy!
Mouse lung large airway (cell nuclei blue) transduced with an adenoviral vector (green).
Purifying mRNA from tissue samples.
A pellet of E.coli containing a plasmid expressing a pink fluorescent protein.